DEFERASIROX GASTROINTESTINAL TOLERABILITY STUDY

• Research type

  Research Study

• Full title

  A phase IV, open-label, partial cross-over partial parallel, randomized, multi-centre study to compare the gastrointestinal tolerability of once daily oral deferasirox, when administered before or after food in patients with transfusional haemosiderosis.

• IRAS ID

  60609

• Contact name

  John B Porter

• Eudract number

  2010-023217-61

• Research summary

  Repeated blood transfusions are necessary in patients with certain types of anaemia such as Beta-Thalassaemia major and other rare anaemias. Repeated transfusions can cause a build-up of excess iron in the body (iron overload) as blood contains iron and the human body does not have a natural way of eliminating excess iron. If untreated iron overload leads to damage to vital organs such as the heart, liver and pancreas and is the leading cause of death and morbidity. Deferasirox is an orally active iron chelator, which removes the excess iron from the body. Deferasirox is licensed by the EMA for the treatment of transfusional iron overload as a once daily dispersible oral tablet. The tablets are dissolved in water, orange juice or apple juice to make a suspension which is taken 30 minutes before food. Current licensing is based on phase II and III studies where the suspension was administered 30 minutes before breakfast. Studies show that about 26% of patients experience gastrointestinal (GI) side effects in the first year of treatment. Clinical experience suggests that administration after food may have better GI tolerability, but this has not yet been confirmed in systematic studies. This study will compare GI tolerability of deferasirox before or after food, in 64 patients with transfusional iron overload over 2 months of treatment. There will be two treatment arms (before and after food) and two patient cohorts in the study. The naÇîve cohort (patients who have not received Deferasirox) will be randomised, and then followed up in parallel design. The non-naÇîve cohort (patients who have been on Deferasirox for over 1 year) will be randomised to a treatment arm and then crossed over to the other arm after a month. This is a two year, multi-centre study being conducted at University College Hospital and Whittington Hospital

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  12/LO/0059

• Date of REC Opinion

  25 Jan 2012

• REC opinion

  Favourable Opinion